What Challenges Did Gene Editing Face in 2018?

TL;DR

In 2018, gene editing, especially CRISPR, encountered significant challenges including delays in clinical trials and concerns over off-target effects and immune responses. Despite some progress, such as moving forward with human trials, the field showed that caution is needed due to potential risks associated with these innovative therapies.

Transcript

Shannon Jones: Let's dive into top story No. 4, and that's related to gene editing. For our listeners, for investors out there, gene editing headlines likely have filled your news feeds over the past year or two, some for good reasons, some for not-so-good reasons. The promise and hype of gene editing, being able to actually go to the underlying ca... Read More

Key Insights

• 🏑 The promise and hype surrounding gene editing have attracted investor enthusiasm, but setbacks in 2018 indicate the challenges of this field.
• 🥹 The delays and regulatory holds experienced by gene editing approaches, such as CRISPR, highlight the need for caution in advancing these treatments.
• 🎯 Concerns about off-target effects, immune system responses, and potential risks like cancer indicate the importance of thorough evaluation and monitoring during clinical trials.
• 🖐️ The zinc finger nuclease approach to gene editing has been studied for a longer time than CRISPR, but disappointing results have also tempered optimism.

Questions & Answers

Q: What are some of the setbacks faced by gene editing in 2018?

Gene editing, particularly the CRISPR approach, experienced delays in clinical trials due to regulatory holds. The first big stumble occurred in May when the FDA delayed a human clinical trial for sickle cell disease. Later, in August, human trials for beta thalassemia were allowed to proceed, but setbacks followed in October and November, causing uncertainty and caution.

Q: What are the concerns related to off-target effects and the immune system's response to gene editing?

There are studies suggesting an increased risk of cancer associated with the CRISPR-Cas9 system, raising concerns about its commercial viability. Additionally, some studies indicate that certain patients may not respond well to CRISPR and may experience an immune response stronger than necessary, potentially impacting safety and efficacy.

Q: How are regulators expected to respond to gene editing companies going through clinical trials?

As the first human trials of gene editing approaches get underway, regulators are expected to closely scrutinize these companies. The pathway to approval will likely be heavily monitored and evaluated to ensure safety and efficacy.

Q: Are there other gene editing approaches besides CRISPR?

Yes, besides the widely known CRISPR-Cas9 approach, there is another gene editing approach called zinc finger nuclease. Studies for this approach have been ongoing since the early 2000s. However, disappointing results were reported in September 2018 for a gene editing approach targeting Hunter syndrome, dampening enthusiasm for this method as well.

Summary & Key Takeaways

• Gene editing shows promise in addressing the underlying cause of genetic diseases before they occur, but 2018 saw several setbacks and challenges.

• Clinical trials for gene editing approaches, including CRISPR, faced delays and setbacks, causing stutter steps in progress.

• Studies indicate potential risks of off-target effects and immune system responses to gene editing treatments.